The National Heart, Lung, and Blood Institute (NHLBI) awarded a $320,251 Project Grant (CFDA 93.837 Cardiovascular Diseases Research) to Ogb5, Inc. for the development of a non-viral nanoparticle gene therapy delivery platform to treat cystic fibrosis and other chronic lung diseases. The project aims to demonstrate that Ogb5's proprietary DNA and RNA nanoparticle technology, combined with its library of enhancer molecules, can effectively deliver therapeutic gene loads and restore primary...
This $783,000 Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), aims to develop a novel non-viral base editing platform for treating cystic fibrosis (CF) caused by nonsense mutations. The key objectives are: 1) Refining RNA-encoded adenine base editors (ABEs) to optimize on-target gene editing; 2) Developing lipid nanoparticle (LNP) formulations for pulmonary delivery; and 3)...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $2,368,153 Project Grant (CFDA 93.837 - Cardiovascular Diseases Research) to The Johns Hopkins University to develop an adeno-associated viral (AAV) vector-based gene therapy for cystic fibrosis (CF). The project aims to evaluate the safety and efficacy of the AAV1-D27-264CFTR vector in non-human primate and ferret models, as well as conduct a Phase I clinical trial in patients with the F508 CF mutation. The research seeks to...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $1,845,871 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to the University of Kansas Medical Center Research Institute, Inc. The grant aims to develop novel gene editing therapies for cystic fibrosis (CF) using a CRISPR-based approach called Homology-Independent Targeted Integration (HITI). The key objectives are to rebuild a mutation-free cystic fibrosis transmembrane conductance regulator (CFTR)...
This federal Project Grant award for $300,000 from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) supports research by Gigamune, Inc. to develop an in vivo gene therapy for treating sickle cell disease. The project aims to test a novel lentiviral vector technology to efficiently edit hematopoietic stem cells and deliver CRISPR/Cas9 machinery to knockout the BCL11A enhancer, with the goal of long-term production of healthy...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) provides $805,845 to the Proteogenomics Research Institute for Systems Medicine to develop and test a novel lung precision drug delivery system (LPDDS) targeting lung myofibroblasts. The goal is to actively transport anti-fibrotic therapies directly into lung tissue to comprehensively block activation and differentiation of myofibroblasts,...
The National Heart, Lung, and Blood Institute (NHLBI) awarded Clemson University a $580,863 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to develop and optimize silicon nanowire-engineered human cardiac organoids for heart repair. The key products and services being delivered include: Determining the optimal surface roughness of electrically conductive silicon nanowires to improve their interactions with human pluripotent stem cell-derived cardiomyocytes in...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $483,852 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to The General Hospital Corporation, doing business as Massachusetts General Hospital (MGH), to develop a high-throughput platform technology to measure the equilibrium and kinetics of sickle hemoglobin polymerization in single red blood cells. The goal of this 2-year research project is to create a novel measurement platform that can quantify...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $486,291 Project Grant (CFDA 93.837 - Cardiovascular Diseases Research) to Rutgers University-Camden to enhance genome editing in hematopoietic stem cells using nucleic acid nanoparticle (NANP) technology. The overall goal is to develop NANP-based delivery methods to more efficiently deliver the CRISPR/Cas9 gene editing system into hematopoietic stem cells, improving targeting precision and efficacy for treating genetic blood...
This $750,672 Project Grant awarded by the National Heart Lung and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) supports research into identifying the cellular protein quality control mechanisms that impact the trafficking and degradation of the CFTR protein variant associated with cystic fibrosis. The research aims to fill critical knowledge gaps and advance new therapeutic targets for treating cystic fibrosis patients who do not respond to existing...